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A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

Primary Purpose

Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome

Status
Completed
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
laronidase
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis I

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-L-iduronidase activity (<10% of the lower limit of normal). For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion. Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years. Exclusion Criteria: Have previously received Aldurazyme without the collection of baseline samples as specified. Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution. Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant. Are receiving chronic immunosuppressant therapy. Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities. Are pregnant or lactating Have received investigational drug within 30 days prior to study enrollment

Sites / Locations

  • Childrens Hospital Los Angeles
  • Connecticut Children's Medical Center
  • Children's Hospital of Wisconsin

Outcomes

Primary Outcome Measures

Urinary GAG (glycosaminoglycans)
Immunogenicity Testing

Secondary Outcome Measures

Safety

Full Information

First Posted
September 2, 2005
Last Updated
May 2, 2014
Sponsor
Genzyme, a Sanofi Company
Collaborators
BioMarin/Genzyme LLC
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1. Study Identification

Unique Protocol Identification Number
NCT00144768
Brief Title
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Official Title
A Multicenter, Multinational, Open-Label Study of Anti-Laronidase Antibody Formation and Urinary GAG Levels in Patients With Mucopolysaccharidosis I (MPS I) Being Treated With Aldurazyme® (Laronidase).
Study Type
Interventional

2. Study Status

Record Verification Date
April 2013
Overall Recruitment Status
Completed
Study Start Date
July 2004 (undefined)
Primary Completion Date
May 2007 (Actual)
Study Completion Date
May 2007 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company
Collaborators
BioMarin/Genzyme LLC

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Anticipated)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
laronidase
Other Intervention Name(s)
Recombinant Human Alpha-L-Iduronidase, Aldurazyme®
Intervention Description
dose of 0.58mg/kg body weight IV every week
Primary Outcome Measure Information:
Title
Urinary GAG (glycosaminoglycans)
Time Frame
Up to 4 years
Title
Immunogenicity Testing
Time Frame
Up to 4 years
Secondary Outcome Measure Information:
Title
Safety
Time Frame
Up to 4 years

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Have a documented diagnosis of MPS I confirmed by measurable signs and symptoms and deficient α-L-iduronidase activity (<10% of the lower limit of normal). For a patients receiving Aldurazyme therapy prior to study entry: Have available as baseline data the results of urinary GAG levels and IgG antibody titers collected prior to the patient's first Aldurazyme infusion. Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years. Exclusion Criteria: Have previously received Aldurazyme without the collection of baseline samples as specified. Have a suspected hypersensitivity to Aldurazyme or a know hypersensitivity to components of infusion solution. Have received a Hematopoietic Stem Cell Transplantation, injection fibroblast therapy, or major organ transplant. Are receiving chronic immunosuppressant therapy. Have a medical condition, serious intercurrent illness, or other extenuating circumstances that may interfere with study compliance including all prescribed evaluations and follow-up activities. Are pregnant or lactating Have received investigational drug within 30 days prior to study enrollment
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
Facility Name
Childrens Hospital Los Angeles
City
Los Angles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Connecticut Children's Medical Center
City
Hartford
State/Province
Connecticut
ZIP/Postal Code
06106
Country
United States
Facility Name
Children's Hospital of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States

12. IPD Sharing Statement

Learn more about this trial

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

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